Welcome to Gene Therapy Net
Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.
First gene therapy to treat rare blood disease nears European approval
Posted on: 2 April 2019, source: STATnews
The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency. A final approval decision is expected within the next three months.
The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency. A final approval decision is expected within the next three months.
Should gene therapy patients be asked to pay?
Posted on: 28 March 2019, source: evaluate.com
The price in value-based payment deals should by definition be justified, so why is patient cost-sharing part of the calculation? Spark covers this cost, but there is no guarantee that all gene therapy companies will. As Novartis’s gene therapy Zolgensma nears a US FDA decision, the sector has only a vague idea of how much the Swiss group will charge for the spinal muscular atrophy (SMA) treatment, other than it will be a number followed by six zeroes. Just as importantly, the families with children suffering from this fatal condition do not know what, if anything, they will be asked to pay. However, there are some clues from the way in which the first US-approved gene therapy, Spark Therapeutics’ Luxturna, was financed.
The price in value-based payment deals should by definition be justified, so why is patient cost-sharing part of the calculation? Spark covers this cost, but there is no guarantee that all gene therapy companies will. As Novartis’s gene therapy Zolgensma nears a US FDA decision, the sector has only a vague idea of how much the Swiss group will charge for the spinal muscular atrophy (SMA) treatment, other than it will be a number followed by six zeroes. Just as importantly, the families with children suffering from this fatal condition do not know what, if anything, they will be asked to pay. However, there are some clues from the way in which the first US-approved gene therapy, Spark Therapeutics’ Luxturna, was financed.
New Gene Therapy Shows Promise For Patients With Sickle Cell Disease
Posted on: 9 March 2019, source: CBS News
An experimental and innovative type of gene therapy is being used to attempt to cure sickle cell anemia. The director of the National Institutes of Health says the results of an NIH clinical trial on a gene therapy for sickle cell anemia "looks like a cure" for the painful and often deadly genetic disease that affects 100,000 Americans, with nearly all being African Americans. 60 Minutes follows the trial for more than a year, focusing on a Florida woman who now appears to be rid of sickle cell anemia. Dr. Jon LaPook's report will be broadcast on 60 Minutes, Sunday, March 10 at 7:00 p.m. ET/PT on CBS.
An experimental and innovative type of gene therapy is being used to attempt to cure sickle cell anemia. The director of the National Institutes of Health says the results of an NIH clinical trial on a gene therapy for sickle cell anemia "looks like a cure" for the painful and often deadly genetic disease that affects 100,000 Americans, with nearly all being African Americans. 60 Minutes follows the trial for more than a year, focusing on a Florida woman who now appears to be rid of sickle cell anemia. Dr. Jon LaPook's report will be broadcast on 60 Minutes, Sunday, March 10 at 7:00 p.m. ET/PT on CBS.
Gene Therapy Drug Approval to Skyrocket by 2025
Posted on: 16 January 2019, source: FDA
The FDA expects to approve 10 to 20 cell and gene therapy products a year within the next six years, and is hiring 50 more clinical reviewers to make it happen, the agency announced Jan. 15: "The FDA is witnessing a surge of cell and gene therapy products entering early development, evidenced by a large upswing in the number of investigational new drug (IND) applications. Based on this activity, we anticipate that the number of product approvals for cell and gene therapies will grow in the coming years, reflecting significant scientific advancement and the clinical promise of these new innovations."
The FDA expects to approve 10 to 20 cell and gene therapy products a year within the next six years, and is hiring 50 more clinical reviewers to make it happen, the agency announced Jan. 15: "The FDA is witnessing a surge of cell and gene therapy products entering early development, evidenced by a large upswing in the number of investigational new drug (IND) applications. Based on this activity, we anticipate that the number of product approvals for cell and gene therapies will grow in the coming years, reflecting significant scientific advancement and the clinical promise of these new innovations."
Gene therapy for Crigler-Najjar syndrome - First patient treated in the European clinical trial
Posted on: 25 December 2018, source: Eurekalerts
Following patient recruitment and preliminary observation period, the first patient was injected by Prof. Labrune at Beclere Hospital in Clamart, France. Genethon, the AFM-Telethon laboratory, is sponsoring this multicenter international phase I/II trial, injecting intravenously an AAV vector that is able to transfer the UGT1A1 gene (coding for the production of bilirubin GT) into liver cells. A total of 17 patients will be treated over the next few months.
Following patient recruitment and preliminary observation period, the first patient was injected by Prof. Labrune at Beclere Hospital in Clamart, France. Genethon, the AFM-Telethon laboratory, is sponsoring this multicenter international phase I/II trial, injecting intravenously an AAV vector that is able to transfer the UGT1A1 gene (coding for the production of bilirubin GT) into liver cells. A total of 17 patients will be treated over the next few months.
Chinese Researchers Claim to Have Genetically Engineered the First HIV-Immune Babies
Posted on: 28 November 2018, source: fortune.com
Researchers in China (Clinical project 'Safety and validity evaluation of HIV immune gene CCR5 gene editing in human embryos') used genetic engineering tools (CRISPR) to create twins theoretically immune to HIV, smallpox, and cholera, MIT Technology Review reported. The medical breakthrough is controversial, as many worry about eugenics and designer babies for the wealthy. The twins, named Lulu and Nana, according to lead scientist He Jiankui of Shenzhen in a YouTube video, were the result of in vitro fertilization (IVF). A few weeks old, they appear to be healthy. When they were a single cell, genetic surgery using a popular tool, CRISPR, “removed the doorway through which HIV enters to infect people.”
Researchers in China (Clinical project 'Safety and validity evaluation of HIV immune gene CCR5 gene editing in human embryos') used genetic engineering tools (CRISPR) to create twins theoretically immune to HIV, smallpox, and cholera, MIT Technology Review reported. The medical breakthrough is controversial, as many worry about eugenics and designer babies for the wealthy. The twins, named Lulu and Nana, according to lead scientist He Jiankui of Shenzhen in a YouTube video, were the result of in vitro fertilization (IVF). A few weeks old, they appear to be healthy. When they were a single cell, genetic surgery using a popular tool, CRISPR, “removed the doorway through which HIV enters to infect people.”
Watch the presentation of He Jiankui and discussion during Second International Summit on Human Genome Editing in Hongkong, Wednesday November 28th, 2018.
Spark Therapeutics' Luxturna Gets EU Approval for Vision Loss
Posted on: 26 November 2018, source: Nasdaq
Spark Therapeutics, Inc. announced that the European Commission has approved the marketing authorization application ("MAA") for its gene therapy, Luxturna (voretigene neparvovec). The MAA sought approval for the therapy in adult as well as pediatric patients as a one-time treatment for vision loss due to inherited retinal dystrophy caused by a genetic mutation in both copies of the RPE65-gene and who have enough viable retinal cells. The RPE65 mutations, a progressive disease, can lead to total blindness.
Spark Therapeutics, Inc. announced that the European Commission has approved the marketing authorization application ("MAA") for its gene therapy, Luxturna (voretigene neparvovec). The MAA sought approval for the therapy in adult as well as pediatric patients as a one-time treatment for vision loss due to inherited retinal dystrophy caused by a genetic mutation in both copies of the RPE65-gene and who have enough viable retinal cells. The RPE65 mutations, a progressive disease, can lead to total blindness.
Gene Therapy News
- Novartis Says Baby's Death May Be Tied to Gene Therapy - Bloomberg
Tue, 23 Apr 2019 06:50 GMT - Pharmalittle: Novartis probes second death in gene therapy trial - STAT
Mon, 22 Apr 2019 13:20 GMT - Asia Regulatory Roundup: Korean Gene Therapy Probe Triggers Accusations of Data Fabrication, Lawsuit - Regulatory Focus
Tue, 23 Apr 2019 17:11 GMT - 'Bubble Boy' Disease Helped By Advances In Gene Therapy : Shots - Health News - NPR
Wed, 17 Apr 2019 21:12 GMT - Gene Therapy for CNS Disorders: Global Market Outlook 2019-2023 with Biogen, Bluebird Bio, Novartis, Pfizer, and Voyager Therapeutics Dominating - ResearchAndMarkets.com - Clearfield Progress
Tue, 23 Apr 2019 11:58 GMT
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You can buy a unique research paper online on gene therapy topics here.